EP4713456A1 - Procédé de modulation d'épissage d'arn - Google Patents

Procédé de modulation d'épissage d'arn

Info

Publication number
EP4713456A1
EP4713456A1 EP24806633.4A EP24806633A EP4713456A1 EP 4713456 A1 EP4713456 A1 EP 4713456A1 EP 24806633 A EP24806633 A EP 24806633A EP 4713456 A1 EP4713456 A1 EP 4713456A1
Authority
EP
European Patent Office
Prior art keywords
mrna
sequence
drna
target
rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP24806633.4A
Other languages
German (de)
English (en)
Inventor
Wensheng Wei
Zongyi YI
Huixian Tang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Peking University
Original Assignee
Peking University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Peking University filed Critical Peking University
Publication of EP4713456A1 publication Critical patent/EP4713456A1/fr
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

Landscapes

  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

L'invention concerne des procédés pour induire un saut d'exon dans un pré-ARNm dans une cellule hôte comprenant l'introduction d'un ARN de recrutement de désaminase (ARNd) ou d'une construction comprenant un acide nucléique codant pour l'ARNd dans la cellule hôte, l'ARNd étant un ARN circulaire (ARNcirc) ou formant un ARNcirc à l'intérieur de la cellule hôte.
EP24806633.4A 2023-05-16 2024-05-16 Procédé de modulation d'épissage d'arn Pending EP4713456A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2023094452 2023-05-16
PCT/CN2024/093632 WO2024235283A1 (fr) 2023-05-16 2024-05-16 Procédé de modulation d'épissage d'arn

Publications (1)

Publication Number Publication Date
EP4713456A1 true EP4713456A1 (fr) 2026-03-25

Family

ID=93518714

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24806633.4A Pending EP4713456A1 (fr) 2023-05-16 2024-05-16 Procédé de modulation d'épissage d'arn

Country Status (3)

Country Link
EP (1) EP4713456A1 (fr)
CN (1) CN121311590A (fr)
WO (1) WO2024235283A1 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121059628A (zh) * 2024-06-03 2025-12-05 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
JP7347830B2 (ja) * 2018-06-29 2023-09-20 エバーハルト カール ウニヴェルジテート テュービンゲン Ｒｎａ編集のための人工核酸
EP3864152B1 (fr) * 2018-10-12 2023-08-16 Peking University Procédés et compositions pour éditer des arn
EP3997229A4 (fr) * 2019-07-12 2024-07-03 Peking University Édition ciblée d'arn par exploitation d'adar endogène à l'aide d'arn modifiés
CN116209762A (zh) * 2020-07-30 2023-06-02 阿达尔克斯制药有限公司 Adar依赖性编辑组合物及其使用方法
WO2022078569A1 (fr) * 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Acides nucléiques artificiels pour édition d'arn
WO2022087451A1 (fr) * 2020-10-23 2022-04-28 The Broad Institute, Inc. Nucléases guidées par acide nucléique et utilisation associée
JP2024504608A (ja) * 2021-01-12 2024-02-01 北京大学 遺伝子操作ｒｎａを使用した内因性ａｄａｒの活用による標的化ｒｎａの編集
EP4370680A4 (fr) * 2021-07-16 2025-08-13 Aptah Bio Inc Compositions polynucléotidiques et procédés de régulation de l'expression génique

Also Published As

Publication number Publication date
CN121311590A (zh) 2026-01-09
WO2024235283A1 (fr) 2024-11-21

Similar Documents

Publication Publication Date Title
US11661596B2 (en) Targeted RNA editing by leveraging endogenous ADAR using engineered RNAs
US20240132890A1 (en) Nucleic acid molecules for pseudouridylation
EP3288594B1 (fr) Système de vecteur aav double pour la correction médiée par crispr/cas9 d'une maladie humaine
EP3116997B1 (fr) Méthodes et compositions associées aux crispr/cas, utilisées dans le traitement de l'amaurose congénitale de leber 10 (lca10)
KR102551664B1 (ko) 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
CA3173012A1 (fr) Compositions et procedes utilisant des composants de snarn
US20130281516A1 (en) Multicistronic expression constructs
US20240309368A1 (en) Targeted rna editing by leveraging endogenous adar using engineered rnas
JP7755272B2 (ja) 操作されたａｄａｒ動員ｒｎａ及びその使用方法
US20230096378A1 (en) Composition for inducing apoptosis of cells having genomic sequence variation and method for inducing apoptosis of cells by using composition
US20240398990A1 (en) Liver-specific expression cassettes, vectors and uses thereof for expressing therapeutic proteins
US20200263206A1 (en) Targeted integration systems and methods for the treatment of hemoglobinopathies
WO2024235283A1 (fr) Procédé de modulation d'épissage d'arn
WO2022182835A1 (fr) Compositions et méthodes d'amélioration de la thérapie génique
JP7575761B2 (ja) 核酸封入ａａｖ中空粒子の製造方法
WO2024255817A1 (fr) Systèmes et méthode pour inhiber la dégradation non-sens avec des arn circulaires
WO2025056035A1 (fr) Compositions et méthodes de traitement de la dystrophie musculaire de duchenne
WO2023185231A1 (fr) Arn de recrutement adar modifiés et procédés d'utilisation pour le syndrome d'usher
WO2023143539A1 (fr) Arn de recrutement d'adar modifiés et leurs procédés d'utilisation
WO2025186392A1 (fr) Édition de génome abca4 thérapeutique pour le traitement d'une maladie stargardt
WO2025193808A1 (fr) Thérapie de remplacement de gène spécifique au snc pour des formes génétiques de la sla/ftd
JP2024529279A (ja) Ｒｂｍ２０変異のゲノム編集
CN121263523A (zh) 核酸关闭开关及其方法和用途

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20251215

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC ME MK MT NL NO PL PT RO RS SE SI SK SM TR